The J. David Gladstone Institutes will work with iZumi Bio Inc. on using adult stem cells in cardiovascular treatments. This research will focus on so-called "induced pluripotent stem cells," which are adult cells that have been "reprogrammed" to function the way the more controversial embryonic stem cells do. Under this agreement, iZumi will license some Gladstone patents. Deepak Srivastava, M.D., is director of the Gladstone Institute for Cardiovascular Disease. He'll take a seat on iZumi's scientific advisory board as part of the deal.
Thane Kreiner is CEO of iZumi Bio, which is backed by Kleiner Perkins Caufield and Byers and Highland Capital Partners. The company started last year and is based in San Francisco. It's moving to larger space in Mountain View next month. Kreiner worked previously at Affymetrix Inc. (NASDAQ: AFFX). Gladstone, led by President Robert Mahley, M.D., is located in Mission Bay in San Francisco.
Six blind patients have had their eye-sight restored after undergoing pioneering stem cell transplants.
Researchers at Moorfields Eye Hospital in London treated the patients as part of a clinical trial on patients who have lost their sight from chemical accidents or a rare genetic disease. Using stem cells from tissue donors, surgeons grew the cells in the laboratory before transplanting them onto the patients' eyes. Dr Julie Daniels, who is leading the research team, will present the results at a conference on regenerative medicine being held in Welwyn Garden City, Hertfordshire, today. She said: "Before the surgery the patients were barely able to recognise when someone was waving a hand in front of their face but we have restored their vision to the point they can read three to four lines down the eye chart."
Nineteen patients have now received the treatment, known as limbal stem cell therapy, at Moorfields Eye Hospital. The patients were chemical burn victims or sufferers of a rare genetic disease known as aniridia. They had injuries to the limbal cells in their eyes, which are under the eye lid and maintain the transparent layer on the outside of the cornea. Dr Daniels said: "Their cornea becomes opaque, blood vessels grow across it and their eyes become inflamed and they can't see anymore. It is very painful. "By replacing the limbal stem cells, the cornea begins to clear up as the cells are replaced with the healthy transparent layer again. "We can't restore sight completely yet as the material we are growing the stem cells on is slightly opaque, but patients are certainly reporting an improvement."
Ten patients were given the transplant 32 months ago and six of those have showed remarkable recovery. The remaining nine patients were treated eight months ago and are still to have their recovery assessed. Scientists at Moorfields Eye Hospital are also hoping to use stem cells to treat other causes of blindness by creating small patches of retina cells, which detect light at the back of the eye. The Royal National Institute for the Blind (RNIB) welcomed the results of the limbal cell trial. Barbara McLaughlin, campaigns manager at RNIB, said: "Anything that can restore sight to people who thought they had irretrievably lost their eyesight is a major step forward. "This research is very exciting, but we would caution that these treatments can take a significant amount of time and research before they can be widely used." More than 250,000 people suffer injuries to their eyes every year as a result of accidents, although only a small proportion of these are due to burns.
In an approach that could become a new treatment for the 10 to 20 percent of people whose broken bones fail to heal, researchers at the University of North Carolina at Chapel Hill have shown that transplantation of adult stem cells can improve healing of fractures.Adult stem cells are specialized cells with the ability to regenerate tissue in response to damage. However, many patients lack sufficient numbers of these cells and thus cannot heal properly. Researchers have used adult stem cells in a few cases to improve fracture healing, but further studies were needed to show that this method was truly effective and safe before it can be pursued as a new treatment. Now scientists at UNC have provided the scientific foundation for future clinical trials of this approach by demonstrating in animal models that these cells can be used to repair broken bones. "This finding is critical to patients who lack the proper healing process and to individuals prone to broken bones, such as those with osteoporosis and the rare genetic condition known as brittle bone disease," said Dr. Anna Spagnoli, associate professor of pediatrics and biomedical engineering in the UNC School of Medicine and senior author on the study.
The study, presented June 16 at the annual Endocrine Society meeting in San Francisco by the first author, Froilan Granero-Molto, Ph.D., post-doctoral associate researcher in UNC's pediatrics department, is the first to visualize the action of transplanted adult stem cells as they mend fractures in mice. During normal fracture healing, stem cells migrate to the site of the break, forming the cartilage and bone needed to fuse the broken bones back together. But in more than 600,000 Americans a year, this process does not occur as it should and these bones stay broken. The result can be long periods of immobilization, pain, bone deformities and even death. Current therapies, such as multiple surgeries with bone autografts and artificial prosthetic materials, often are not enough to cure these patients.
"Man-made materials do not address the normal bone's function, and recurrent fractures, wear and toxicity are a real problem," Spagnoli said. "There is clearly a need to develop alternative therapies to enhance fracture healing in patients with bone union failure." Kicking stem cells into repair mode is one of the objectives of a new branch of medicine called regenerative medicine. With a little prodding, stem cells in human bone marrow -- called mesenchymal stem cells -- can turn into bone, cartilage, fat, muscle and blood vessel cells. "The beauty of regenerative medicine is that we are helping the body improve its innate ability to regenerate healthy tissue on its own, rather than introducing manmade materials to try to patch up a broken bone," Spagnoli said. Granero-Molto and other colleagues led by Spagnoli demonstrated this approach by transplanting adult stem cells in mice with fractures of the tibia, the long bone of the leg. The cells were taken from the bone marrow of mice that produce luciferase, the same molecule that allows fireflies to glow. In addition to possessing the ability to glow, the cells were engineered to express a molecule called insulin-like growth factor 1 (IGF-1). IGF-1 is a potent bone regenerator necessary for bones to grow both in size and strength.
The researchers transplanted the cells through a simple intravenous injection and then placed the mice into a dark box so they could track the glowing stem cells as they migrated within the rodent. They found that these cells were specifically attracted to the fracture site, and that a particular molecule called CXCR4 -- which acts as a homing signal -- was necessary for the migration. Using a computerized tomography (CT or CAT) scan, the researchers showed that the stem cells not only migrated to the site of the fracture, but also improved healing there by increasing the bone and cartilage that bridged the bone gap. The bone at the fracture site in the treated mice was about three times stronger than that of untreated controls. If scientists can duplicate the results of this animal study in humans, it may lead to a new treatment for the millions of people who suffer fractures that do not heal properly, Spagnoli said. Once a physician determines that the bone has not healed, they could obtain adult stem cells from the person's bone marrow in a minimally invasive procedure and transplant them at the same time the patient is receiving a bone graft.
Spagnoli said adult stem cells may pose fewer problems than embryonic stem cells, since they are not associated with the ethical controversy that surrounds the latter. Also, they may avoid the problem of rejection by the immune system, since the patient's own cells can be used. Funding for the study came from the National Institutes of Health. Other co-authors of the study include Dr. Lara Longobardi, UNC assistant professor of pediatrics, along with the following researchers from Vanderbilt University: Dr. Michael Miga, assistant professor of biomedical engineering; Dr. Jared A. Weis, postgraduate fellow in biomedical engineering; Benjamin Landis, medical student; and Lynda O'Rear, research specialist.